Cystic Fibrosis Lung study: 4D-710
Official title 4D-710 in Adult Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT05248230
What this study is testing
What is 4D-710?
4D-710 is an investigational gene therapy being studied as a potential treatment for cystic fibrosis lung disease. It is designed to deliver a corrected copy of the CFTR gene into lung cells using a modified virus.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to evaluate the safety and effectiveness of a single dose of 4D-710 gene therapy in adults with cystic fibrosis.
- Who it's for
- Adults diagnosed with cystic fibrosis.
- What's involved
- Participants receive one dose of the investigational gene therapy and attend follow‑up visits for monitoring and testing.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 18 years and older
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease
You likely can't join if
- Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
- Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
- Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
- Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening
- Contraindication to systemic corticosteroid therapy
- Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
See the full eligibility criteria
- 18 years and older
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease
- Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects.
- Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening
- 18 years and older
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease
- Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period
- FEV1 ≥ 40% and \< 70% predicted (per Global Lung Function Initiative) at Screening, AND/OR experienced at least 2 pulmonary exacerbations in the last year requiring intravenous antibiotics Key
- Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
- Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
- Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
- Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening; must agree to not smoke or use vaping products during Screening and initial 24-month observation period.
- Contraindication to systemic corticosteroid therapy
- Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
- If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening
- If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C \> 7.5% at Screening
- Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment
- Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia
- Body Mass Index (BMI) \< 16
- ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
- Total bilirubin ≥ 2 × ULN
- Hemoglobin \< 10 g/dL
- Requirement for continuous or night-time oxygen supplementation
- Known CF liver disease with evidence of multilobular cirrhosis
- History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
The study team makes the final eligibility decision.
Where it is enrolling
- Birmingham, Alabama, United States
- Tucson, Arizona, United States
- San Francisco, California, United States
- Denver, Colorado, United States
- Gainesville, Florida, United States
- Miami, Florida, United States
- Chicago, Illinois, United States
- Kansas City, Kansas, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Charleston, South Carolina, United States
- Dallas, Texas, United States
- Richmond, Virginia, United States
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Birmingham, Alabama, United States; Tucson, Arizona, United States; San Francisco, California, United States; Denver, Colorado, United States; Gainesville, Florida, United States; Miami, Florida, United States and 11 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.